Solid Biosciences announced today that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne.

The Phase I/II adaptive study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous (IV) dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with Duchenne. Enrollment will begin at the first clinical trial site in the United States in the coming days.

You can read both the press release and community letter from Solid below. PPMD is excited to see companies continuing to explore potential therapies like microdystrophin gene transfer to treat Duchenne.

Solid's Letter To Duchenne Community:

Dear Duchenne community members,

Today we are excited to announce that we are initiating our first clinical trial for SGT-001, our investigational microdystrophin gene transfer for Duchenne muscular dystrophy. This milestone is a result of years of effort, including extensive collaboration with the top experts in gene therapy and DMD and the completion of our preclinical program. It also reflects the ongoing support and insights the DMD community has generously shared with us.

The Phase I/II clinical trial, called IGNITE DMD, is a randomized, controlled, open-label, single-ascending dose study that will evaluate the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients with DMD.

IGNITE DMD, adaptive in nature, will allow Solid Biosciences to adjust dose and number of patients as the study progresses to efficiently characterize the safety and efficacy of SGT-001.

Patient screening will begin at our first participating study location in the United States in the coming days. We are working to bring on additional sites in the United States and abroad. We want to highlight a few important details about the study:

IGNITE DMD will enroll non-ambulatory and ambulatory males with DMD between the ages of 4 and 17.

We anticipate enrolling approximately 16 to 32 patients.

There is no restriction based on a patient’s underlying dystrophin gene mutation.

All participants must have stable cardiac and respiratory function.

All participants must be below a pre-determined threshold for antibodies against adeno-associated virus (AAV), which must be tested as part of the screening process at the study sites. To learn more about AAV, click here to view our gene therapy education program.

IGNITE DMD will evaluate two groups of patients: an active treatment group in which patients will receive a single IV dose of SGT-001 and a delayed-treatment group. By following the active and delayed treatment groups in parallel, we believe we can more efficiently and effectively determine if any benefits or adverse events in the study are due to SGT-001 treatment. After 12 months, patients in the control group who still meet treatment criteria will receive SGT-001. Assignment of patients to the active and control groups will be done randomly using a computer, and treatment assignment will be fully transparent to participating patients and families.

If you are interested in learning more about IGNITE DMD, further information will be available on clinicaltrials.gov shortly. There you will find additional details on the inclusion and exclusion criteria, as well as how to contact the sites participating on this study. Patient screening and selection will be handled by investigators at the clinical sites; members of the Solid team are not involved in those decisions.

The initiation of IGNITE DMD marks an important step forward in our mission to develop meaningful treatments that will improve the lives of as many patients with DMD as possible. However, this moment is as sobering as it is exciting. While data from our preclinical program are strong, systemic gene transfer for DMD is still a novel technology and we may not yet know all its potential risks and benefits. Our clinical trial will begin to provide us with answers to these questions.

We recognize how important information is to the community. As we move forward in this journey, we promise to share relevant information and program updates whenever we are able.

-Phase I/II adaptive clinical trial to evaluate safety and efficacy in ambulatory and non-ambulatory children and adolescents-

-Interim analysis anticipated in the first half of 2019-

Cambridge, MA, November 30, 2017 -- Solid Biosciences announced today that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne muscular dystrophy (DMD). The Phase I/II adaptive study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous (IV) dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with DMD. Enrollment will begin at the first clinical trial site in the United States in the coming days.

“For more than three years, we’ve been working on a preclinical package and scalable manufacturing process for SGT-001 that would allow us to responsibly move into the clinic,” said Ilan Ganot, Founder and Chief Executive Officer of Solid Biosciences. “This work, as well as decades of research by our scientific advisors and insight from the DMD community, helped us design a clinical trial that will efficiently characterize the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients, regardless of their underlying genetic mutation.”

IGNITE DMD, a single-ascending dose study, is adaptive in nature, which will allow Solid to adjust the dose and number of participants as the study progresses to efficiently assess the safety and efficacy of SGT-001. Solid expects to enroll approximately 16 to 32 patients in the study, all of whom will receive a systemic dose of SGT-001. The starting dose was selected based on Solid’s extensive preclinical program across multiple animal species. All clinical drug product is being produced utilizing Solid’s scalable manufacturing process.

Solid is initiating its study under an Investigational New Drug (IND) application cleared by the U.S. Food and Drug Administration (FDA) on October 12, 2017. A pre-specified interim analysis of preliminary data is planned and will be communicated in the first half of 2019.

“For more than 20 years, my team has focused our research on understanding the potential of microdystrophin in DMD and optimizing a construct that we believe will provide the most benefit for patients,” said Dr. Jeffrey Chamberlain, Professor, Department of Neurology; Professor, Department of Biochemistry; and Professor of Medicine, Division of Medical Genetics at the University of Washington. “I’m pleased to see Solid advance the most compelling of constructs into clinical studies. I believe we are now at an inflection point where, if successful, SGT-001 should represent an innovative new way of treating patients with this devastating disease.”